SUN-2019-REGO-BTC

Regimen

Experimental
Regorafenib 160 mg orally daily × 21 days of a 28-day cycle until progression
Control
single-arm / no comparator

Population

Advanced BTC (unresectable/metastatic) refractory to ≥1 prior gemcitabine-based chemotherapy; ICC ~53%, ECC ~23%, GBC ~24%; US multicenter (University of Pittsburgh and collaborators); ECOG PS 0-1; all prior GemCis or GemOx

Key finding

Regorafenib showed modest single-agent activity in refractory BTC with a 56% DCR but only 11% ORR and a short median PFS of 2 months. The 7.4-month median OS is consistent with 2L BTC historical controls. Grade 3-4 adverse events occurred in ~60% (hand-foot syndrome, fatigue, hypertension — typical regorafenib toxicity profile). The results are exploratory and the trial was not powered for formal efficacy testing. Biomarker correlates (RAS/RAF mutations, VEGFR expression) were explored but no predictive biomarker emerged. This trial did not lead to regulatory approval for regorafenib in BTC.

Source: PMID 30561756

Timeline

    Guideline citations

    • NCCN BTC (p.20)